Patient Death Latest Setback for Gene Therapy
A patient's death in a gene therapy clinical trial may raise public concerns.
July 27, 2007 — -- The death of a patient enrolled in the clinical trial of an experimental gene therapy is the third black mark for the treatment approach since 1999 -- and one that doctors fear has the potential to stall future research.
The death has spurred the U.S. Food and Drug Administration (FDA) to halt the trial and begin safety reviews of 28 other gene therapy trials around the country.
According to an FDA press release issued Thursday, the Seattle-based Targeted Genetics Corp., which conducted the trial, informed the FDA of the death July 24.
The trial was designed to test the effectiveness of a treatment for active inflammatory arthritis called tgAAC94.
It is not yet clear whether the drug being used directly led to the patient's death, or whether other causes are to blame. Still, in response to the death, the FDA has launched an investigation of the trial, which began in October 2005 and involved 127 subjects who may have received the drug.
According to the FDA statement, none of the other subjects are reported to have experienced similar complications, and the patient's illness was related in time to the receipt of a second injection of the product.
But gene therapy researchers say they are already worried that the death could represent a major setback for the technology, regardless of the results of the investigation.
"Gene therapy may be an important future effort, and it is disconcerting that something like this has happened," said Dr. Lee Simon, associate clinical professor of medicine at Harvard Medical School, who called the announcement "interesting and troubling."
Other doctors agreed.
"It is certainly a troubling development," said Dr. David Fox, chief of rheumatology at the University of Michigan Medical School. "It will be important to determine what caused this patient's death and whether it was due to the gene therapy treatment."
Dr. Michael Becker, a rheumatologist at the University of Chicago Medical Center, said that his first reaction to news of the trial was "sadness at the death of the patient and concern about its implications relevant to gene therapy."
